Recognizing and avoiding siRNA off-target effects for target identification and therapeutic application

  title={Recognizing and avoiding siRNA off-target effects for target identification and therapeutic application},
  author={Aimee L Jackson and Peter S. Linsley},
  journal={Nature Reviews Drug Discovery},
Small interfering RNAs (siRNAs) are widely used to study gene function owing to the ease with which they silence target genes, and there is considerable interest in their potential for therapeutic applications. In a remarkably short time since their discovery, siRNAs have entered human clinical trials in various disease areas. However, rapid acceptance of the use of siRNAs has been accompanied by recognition of several hurdles for the technology, including a lack of specificity. Off-target… 

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  • A. Aigner
  • Biology, Chemistry
    Journal of biomedicine & biotechnology
  • 2006
An overview of studies based on the systemic or local application of naked siRNAs or the use of various nonviral siRNA delivery systems and the antitumorigenic effects of PEI/siRNA-mediated in vivo gene-targeting of tumor-relevant proteins like in mouse tumor xenograft models are described.

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An update on the progress of RNAi therapeutics is provided and novel synthetic materials for the encapsulation and intracellular delivery of nucleic acids are highlighted.

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